Multi-stakeholder sessions on major innovation topics in rare disease clinical trials.

Background: The European Joint Programme on Rare Diseases aims to enhance the rare diseases research ecosystem by bringing together stakeholders such as research funders, institutions and patient organizations. Work Package 20 focuses on the validation, use and development of innovative methodologies for rare disease clinical trials. This paper reports on the outcomes of a retreat held in April 2023, where areas for innovation and educational needs in rare disease clinical trials were discussed in multi-stakeholder sessions.

Cardiac Tumor Within the Left Ventricle: Thrombus or Something Else?

Papillary fibroelastoma is a benign cardiac tumor that most commonly arises from the valvular endocardium and is typically resected because of its predisposition to cause embolic complications. In this clinical vignette, we describe the case of a patient who presented with a round, mobile 1.3 × 1.

Randomized trial of an oblique versus standard fluoroscopic-guided micropuncture technique for femoral arterial access: The Micropuncture-CFA trial.

Background: The anterior-posterior fluoroscopic guidance (the AP technique) is a standard method for common femoral artery (CFA) access, but the rate of CFA access with ultrasound vs. the AP technique was not significantly different. We have shown an oblique fluoroscopic guidance (the oblique technique) with a micropuncture needle (MPN) resulted in CFA access in 100 % of patients.